Galactosemia Voice of the Patients Report

For the first time in history, the Galactosemia community came together for a historic meeting to share their experiences of the disease and its treatments – as well as hopes for the future – with the US Food and Drug Administration (FDA). On September 1, 2022, the Galactosemia Foundation and NORD hosted this externally-led Patient Focused Drug Development (EL-PFDD) meeting, which provided an opportunity for families and caregivers to share valuable insight about the impact of a rare disease on daily life directly with key government and drug developer stakeholders, including researchers, medical product developers, and health care providers.

Nearly 300 attendees engaged live in the dynamic and interactive virtual meeting featuring polling, video remarks, and earnest dialogue among the community. The remarks, presentations, livestream-community chats, and polling have been captured in our  Voice of the Patient Report. There is also an addendum of additional patient and caregiver comments. The report has been submitted to the FDA’s Center for Drug Evaluation and Research (CDER), for inclusion in the framework used to evaluate future therapies. The Galactosemia Foundation and NORD are grateful for engaged partners in the rare disease field interested in hearing patients’ and caregivers’ perspectives – partners who can understand hopes for future treatments, side effects patients are and are not willing to tolerate, the medical risks they are willing to take in clinical trials, and their interests and challenges in participating in those clinical trials.

Download and Read the Galactosemia Voice of the Patient Report

You can also watch the full meeting with FDA below:

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